Every Day With SMA

Navigating life with SMA

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Every Day With SMA

Navigating life with SMA

Quinn proudly stands


A simple yet powerful act

Hear these SMA caregivers discuss how proud their daughter was when she stood for the first time and their hopes for her future.
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JOE: Traditionally, when you think about having a child with SMA, your focus is on the basic necessities of life: eating and breathing and those types of things, but to be in a position where we’re able to see our child stand and improve has completely raised the bar in terms of what our expectations are.

ANNIE: When she received her leg braces and was able to stand for the first time, the look on her face was one of the most incredible moments that I’ve experienced.

JOE: There’s something metaphorically so powerful about someone standing—and what that means—being able to, on their own volition stand, and for us as parents, is amazing to see that physical development.

ANNIE: I know that has to be such an incredible feeling for her to stand and be upright when all she’s known is sitting and laying down.

ANNIE: It made my momma heart just wanna burst.

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Slade, who was diagnosed with SMA Type 1, laying on a blanket and eating a popsicle

Slade’s first popsicle

Watch Slade taste the sweeter side of life in this video commemorating his first popsicle.

Ryker, who was diagnosed with SMA Type 1, in his wheelchair

Ryker turns 2

It's the happiest of birthdays for Ryker, who turned 2 years old! Watch a video of his parents reflecting on this joyous occasion while making lifelong memories.

Preparing for the future

Check out resources and advice from other caregivers to help you navigate your child’s future with SMA.

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Important Safety Information

What is the most important information I should know about ZOLGENSMA?

  • ZOLGENSMA can cause acute serious liver injury. Liver enzymes could become elevated and may reflect acute serious liver injury in children who receive ZOLGENSMA.
  • Patients will receive an oral corticosteroid before and after infusion with ZOLGENSMA and will undergo regular blood tests to monitor liver function.

Important Safety Information

What is the most important information I should know about ZOLGENSMA?

  • ZOLGENSMA can cause acute serious liver injury. Liver enzymes could become elevated and may reflect acute serious liver injury in children who receive ZOLGENSMA.
  • Patients will receive an oral corticosteroid before and after infusion with ZOLGENSMA and will undergo regular blood tests to monitor liver function.
  • Contact the patient’s doctor immediately if the patient’s skin and/or whites of the eyes appear yellowish, or if the patient misses a dose of the corticosteroid or vomits it up.

What should I watch for before and after infusion with ZOLGENSMA?

  • Viral respiratory infections before or after ZOLGENSMA infusion can lead to more serious complications. Contact the patient’s doctor immediately if you see signs of a possible viral respiratory infection such as coughing, wheezing, sneezing, runny nose, sore throat, or fever.
  • Decreased platelet counts could occur following infusion with ZOLGENSMA. Seek immediate medical attention if the patient experiences unexpected bleeding or bruising.
  • Thrombotic microangiopathy (TMA) has been reported to occur approximately one week after ZOLGENSMA infusion. Caregivers should seek immediate medical attention if the patient experiences any signs or symptoms of TMA, such as unexpected bruising or bleeding, seizures, or decreased urine output.

What do I need to know about vaccinations and ZOLGENSMA?

  • Talk with the patient’s doctor to decide if adjustments to the vaccination schedule are needed to accommodate treatment with a corticosteroid.
  • Protection against respiratory syncytial virus (RSV) is recommended.

Do I need to take precautions with the patient’s bodily waste?

Temporarily, small amounts of ZOLGENSMA may be found in the patient’s stool. Use good hand hygiene when coming into direct contact with bodily waste for 1 month after infusion with ZOLGENSMA. Disposable diapers should be sealed in disposable trash bags and thrown out with regular trash.

What are the possible or likely side effects of ZOLGENSMA?

The most common side effects that occurred in patients treated with ZOLGENSMA were elevated liver enzymes and vomiting.

Indication

What is ZOLGENSMA?
ZOLGENSMA is a prescription gene therapy used to treat children less than 2 years old with spinal muscular atrophy (SMA). ZOLGENSMA is given as a one-time infusion into a vein. ZOLGENSMA was not evaluated in patients with advanced SMA.

The safety information provided here is not comprehensive. Talk to the patient’s doctor about any side effects that bother the patient or that don’t go away.

You are encouraged to report suspected side effects by contacting the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch, or Novartis Gene Therapies, Inc. at 833-828-3947.

Please see the Full Prescribing Information.